A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet…
Articles by Larry Luxner
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups…
Swiss pharmaceutical giant Roche plans to begin recruiting this summer for an endpoint study in Angelman syndrome that…
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails…
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves…
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and…
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12…
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the…
G71.01 is, literally, the code for Duchenne muscular dystrophy. Â Q93.51 stands for Angelman syndrome, and G40.419 covers generalized…
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time…